Strategies to facilitate the long-term expression of transgenes for treatment of chronic liver disease

Thesis

The development of new gene therapy products offers ground-breaking opportunities for the treatment of human disease. To provide lasting benefit to patients, the expression of a correct copy of a non-functional gene needs to be life-long, and the maintenance of therapeutic levels of gene expression is a key challenge. Gene therapy for chronic liver disease using recombinant adeno-associated virus, is very encouraging, but as the vector exists as an extra-chromosomal episome, it can be lost...

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Authors: van Haasteren, J

• Type of award: DPhil
• Level of award: Doctoral
• Awarding institution: University of Oxford
• Subjects: Gene editing; Gene therapy